Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing

Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing

Leber congenital amaurosis (LCA), an inherited retinal degeneration, causes severe visual dysfunction in children and adolescents. In patients with LCA, pathogenic variants, such as RPE65, are evident in specific genes, related to the functions of retinal pigment epithelium and photoreceptors. In co...

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Bibliographic Details
Main Authors: Dong Hyun Jo, Hyeon-Ki Jang, Chang Sik Cho, Jun Hee Han, Gahee Ryu, Youngri Jung, Sangsu Bae, Jeong Hun Kim
Format: Article
Language:English
Published: Elsevier 2023-03-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: RNA/DNA editing
base editing
CRISPR
Leber congenital amaurosis
RPE65
adeno-associated virus
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253122003134

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http://www.sciencedirect.com/science/article/pii/S2162253122003134

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